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ClinicalTrials.gov public records Last synced Nov 15, 2026, 4:57 AM EST

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Showing 1–24 of 29 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Ornithine Transcarbamylase Deficiency Clear all

Conditions: Urea Cycle Disorder
Interventions: Acetohydroxamic Acid Oral Tablet, No treatment; Drug · Other
Lead sponsor: Nicholas Ah Mew; Other
Eligibility: 18 Years to 60 Years
Enrollment: 4 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2018
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Urea Cycle Disorders, Organic Acidemias
Interventions: Therapeutic Hypothermia, Standard of Care Therapy; Other
Lead sponsor: Uta Lichter-Konecki; Other
Eligibility: 1 Day to 30 Days
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2015
U.S. locations: 3
States / cities: Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin

Conditions: Ornithine Transcarbamylase (OTC) Deficiency
Interventions: scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen; Genetic · Drug
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 4
States / cities: Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more

Conditions: Maculopathy, Age-Related, Choroidal Neovascularization
Interventions: Not listed
Lead sponsor: Johns Hopkins University; Other
Eligibility: 50 Years and older
Enrollment: 98 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 4
States / cities: Beverly Hills, California • Baltimore, Maryland • Boston, Massachusetts + 1 more

Conditions: Urea Cycle Disorder
Interventions: KB195; Drug
Lead sponsor: Kaleido Biosciences; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Tampa, Florida • Baltimore, Maryland + 3 more

Conditions: Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Not listed
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 2
States / cities: Washington D.C., District of Columbia • Houston, Texas

Conditions: OTC Deficiency
Interventions: DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate; Genetic · Other · Drug
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 12 Years and older
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2031
U.S. locations: 4
States / cities: Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more

Conditions: Urea Cycle Disorder
Interventions: Not listed
Lead sponsor: Amgen; Industry
Eligibility: Not listed
Enrollment: 203 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 1
States / cities: Blue Bell, Pennsylvania

Conditions: Metabolic Diseases
Interventions: human hepatocyte transplantation, Preparative Radiation Therapy; Drug · Radiation
Lead sponsor: Ira Fox; Other
Eligibility: Up to 21 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2022
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Acute Myocardial Infarction, Restenoses, Coronary, Coronary Artery Lesions, Venous Embolism, Arterial Embolism
Interventions: Coronary and peripheral stents, Pacing catheters, Vascular plugs, Measurement and imaging (FFR and OCT), Peripheral dilatation catheters, Coronary dilatation catheters, Coronary and peripheral guidewires, Vessel closure/compression devices, Vascular access introducers; Device
Lead sponsor: Abbott Medical Devices; Industry
Eligibility: 18 Years and older
Enrollment: 3,784 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2031
U.S. locations: 7
States / cities: Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 4 more

Conditions: Argininosuccinic Aciduria, Carbamoyl-Phosphate Synthase I Deficiency, Citrullinemia, Ornithine Carbamoyltransferase Deficiency, Hyperargininemia, N-Acetylglutamate Synthase Deficiency
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 2 Years to 99 Years
Enrollment: 4 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2013
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Ornithine Transcarbamylase Deficiency
Interventions: No Intervention; Other
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Orlando, Florida

Conditions: Optical Coherence Tomography (OCT) of the Retina
Interventions: OCT imaging; Device
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Analgesia
Interventions: Paracetamol, Paracetamol formulation 1, Paracetamol Formulation 2, Paracetamol formulation 3, Paracetamol formulation 4; Drug
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 14 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2008 – 2009
U.S. locations: 1
States / cities: Phoenix, Arizona

Conditions: Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 6 Years to 65 Years
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 5
States / cities: Aurora, Colorado • Washington D.C., District of Columbia • Philadelphia, Pennsylvania + 2 more

Conditions: Ornithine Transcarbamylase Deficiency
Interventions: ARCT-810, Placebo; Biological · Other
Lead sponsor: Arcturus Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 8
States / cities: Gainesville, Florida • Minneapolis, Minnesota • New York, New York + 5 more

Conditions: Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Interventions: Not listed
Lead sponsor: Andrea Gropman; Other
Eligibility: 18 Years to 60 Years
Enrollment: 46 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2007 – 2010
U.S. locations: 2
States / cities: Washington D.C., District of Columbia

Conditions: Coronary Artery Disease
Interventions: OCT stent guidance; Device
Lead sponsor: Abbott Medical Devices; Industry
Eligibility: 18 Years and older
Enrollment: 418 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2015
U.S. locations: 15
States / cities: Birmingham, Alabama • La Jolla, California • Orlando, Florida + 12 more

Conditions: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
Interventions: ECUR-506; Genetic
Lead sponsor: iECURE, Inc.; Industry
Eligibility: 24 Hours to 7 Months · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 6
States / cities: Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 3 more

Conditions: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
Interventions: No Intervention; Other
Lead sponsor: iECURE, Inc.; Industry
Eligibility: 7 Months to 15 Months
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2041
U.S. locations: 2
States / cities: Aurora, Colorado • New York, New York

Conditions: Cancers
Interventions: Laparoscopic surgery; Procedure
Lead sponsor: Erin Rowell; Other
Eligibility: Up to 11 Years · Female only
Enrollment: 250 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2035
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
Interventions: Carbaglu, Placebo; Drug
Lead sponsor: Mendel Tuchman; Other
Eligibility: 1 Week to 99 Years
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2020
U.S. locations: 9
States / cities: Los Angeles, California • Palo Alto, California • Aurora, Colorado + 6 more

Conditions: Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions: Not listed
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Akron, Ohio

Conditions: Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I, Fatty Acid Oxidation Disorder, Hypoxic-Ischemic Encephalopathy
Interventions: Not listed
Lead sponsor: Children's National Research Institute; Other
Eligibility: 7 Years to 18 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Search by objective public record fields.

Manipulating the Gut Microbiome Study

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

Age-related Macular Degeneration: Detection of Onset of New Choroidal Neovascularization (AMD DOC Study)

UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder

Hepatic Histopathology in Urea Cycle Disorders

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)

Hepatocyte Transplantation for Liver Based Metabolic Disorders

Abbott Vascular Medical Device Registry

The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity

Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

Low Cost OCT for Point of Care

A Study Investigating the Pharmacokinetic Profiles of Four Extended Release Paracetamol Formulations

Liver Disease in Urea Cycle Disorders

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Neurologic Injuries in Adults With Urea Cycle Disorders

Observational Study of OCT in a Patients Undergoing FFR

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Systemic Biomarkers of Brain Injury From Hyperammonemia